Avrobio gets orphan drug nod for Fabry disease gene therapy

By The Science Advisory Board staff writers

October 29, 2020 -- Avrobio has been granted orphan drug designation by the European Commission for AVR-RD-01, an ex vivo lentiviral gene therapy that consists of a patient's own hematopoietic stem cells that are genetically modified to express alpha-galactosidase A, the enzyme that is deficient in patients with Fabry disease.

The drug has also received orphan drug designation from the U.S. Food and Drug Administration and is being evaluated in a phase II clinical trial.

Fabry disease is a rare, inherited lysosomal storage disorder characterized by toxic accumulation of globotriaosylceramide in the body due to a variation in the GLA gene.

Avrobio expands Hunter syndrome gene therapy program
Avrobio has signed a research and licensing agreement with the University of Manchester in the U.K. to investigate the use of lentiviral gene therapy...
Avrobio gets orphan drug nod for Gaucher disease gene therapy
Avrobio has been granted orphan drug designation for AVR-RD-02, an investigational gene therapy for treatment of Gaucher disease, by the European Commission.
Avrobio moves forward with gene therapy candidate
Avrobio released preclinical data for AVR-RD-03, lentiviral gene therapy for Pompe disease, at the American Society of Gene and Cell Therapy annual meeting...

Copyright © 2020 scienceboard.net


Conferences
Connect
Science Advisory Board on LinkedIn
Science Advisory Board on Facebook
Science Advisory Board on Twitter